Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

McGrattan, Katlyn; D. Shell, Richard; Hurst-Davis, Rebecca; Dunaway Young, Sally; O’Brien, Eamonn; Lavrov, Arseniy; Wallach, Shiri; LaMarca, Nicole; P. Reyna, Sandra; T. Darras, Basil

doi:10.3233/jnd-221531

Public

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

Published in Journal of Neuromuscular Diseases • Apr 17, 2023

Authors:

Katlyn McGrattan

Richard D. Shell

Rebecca Hurst-Davis

Abstract

BACKGROUND: Improvement and maintenance of bulbar function are goals of disease-modifying treatments for spinal muscular atrophy (SMA). Lack of standardized measures and a widely accepted definition of bulbar function represents a gap in SMA care. OBJECTIVE: A multidisciplinary team conducted post-h...

Subject

Medicine

Swallowing

SMA*

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